FDA officials highlight new "plausible mechanism" pathway and flexibilities for ultra‑rare therapies during Rare Disease Week At a virtual Rare Disease Week town hall, FDA speakers previewed a 'plausible mechanism' pathway for ultra‑rare and bespoke conditions, discussed CMC flexibilities for cell and gene therapies, and emphasized transparency and compassionate‑use access while balancing trial integrity.

FDA officials are unveiling a groundbreaking "plausible mechanism" pathway to accelerate treatments for ultra-rare diseases, promising a brighter future for patients and their families.

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